FDA OKs Acadia’s Rett Syndrome Drug

The long wait for a drug to relive symptoms of Rett syndrome is finally over.

On March 10, San Diego-based Acadia Pharmaceuticals (NASDAQ: ACAD) announced FDA approval of DAYBUE (trofinetide) for treatment of Rett syndrome – a rare neurodevelopmental disorder that affects approximately 6,000 to 9,000 patients in the U.S.

“Today marks an important milestone for the Rett community and Acadia,” said Acadia CEO Steve Davis.  “As the first FDA-approved drug for the treatment of Rett syndrome, DAYBUE now offers the potential to make meaningful differences in the lives of patients and their families who have lacked options to treat the diverse and debilitating array of symptoms caused by Rett syndrome.”

“We are grateful to all of the Rett syndrome patients, caregivers, clinical investigators and our employees who have contributed to making today a reality and look forward to getting DAYBUE to patients as quickly as possible,” Davis added.

Acadia expects DAYBUE to be available for Rett patients as early as late April.

Improvements in ‘Broad’ Rage of Symptoms

Rett syndrome is caused by a genetic mutation and is characterized by a period of normal development until six to 18 months of age, followed by significant developmental regression with loss of acquired communication skills and purposeful hand use. Rett symptoms may also include development of hand stereotypies, such as hand wringing and clapping, and gait abnormalities.

Results from the “robust” Phase III study of DAYBUE were “overwhelmingly positive and demonstrate a compelling benefit profile for patients,” said Kathie Bishop, chief science officer and head of rare diseases at Acadia. “We saw improvement across the broad symptoms of Rhett syndrome.”

Brendan Teehan, chief operating officer and head of commercial at Acadia, said parents of children in the clinical trial would share with him examples of the efficacy of the drug at Rett events Acadia would participate in.

“They would say things like ‘thank you, my daughter is more alert; my daughter used her hands to use the remote control to shoes the show she wanted to watch; she transferred an item from one hand to another; she was able to hold a cup and feed herself,’” he said. “All of these things are an aggregate composite of the types of symptom improvement that we’re hoping to see for as many Rett patients as we possibly can.”

Ensuring Access to Treatment

Acadia’s commitment to improving symptoms of Rett patients includes programs to help families afford the estimated the annual realized cost of DAYBUE of approximately $375,000 per patient.

“Acadia’s patient access philosophy is simple,” Teehan said. “We believe cost should never be a barrier to treatment.”

About 60% of Rett patients are on Medicaid, which will provide the treatment with minimal out-of-pocket expenses. for the 30% on commercial plans, Acadia is providing a co-pay program that will allow “patients and families to pay as little as zero dollars a month,” Teehan said.

To help fill in any remaining gaps in coverage, Acadia will help facilitate funding from foundations and other organizations.

Melissa Kennedy, CEO of the International Rett Syndrome Foundation, described the approval of DAYBUE as “historic” for the Rett syndrome community and “a meaningful moment for the patients and caregivers who have eagerly awaited the arrival of an approved treatment for this condition.”

“Rett syndrome is a complicated, devastating disease that affects not only the individual patient, but whole families,” Kennedy said. “Those impacted by Rett have a promising new treatment option that has demonstrated benefit across a variety of Rett symptoms, including those that impact the daily lives of those living with Rett and their loved ones.”

In addition to the drug treatment, as part of its commercialization strategy Acadia will offer a patient support service hub to include nurse care coordinators that can answer questions for families “before they ever have their child or person they care for start DAYBUE, and understand benefits and side effects, in this case GI tact issues,” Teehan said, adding that the nurse care coordinators as well as Acadia family access managers in the field will be paired with specific families to “create that consistency – a relationship so you can feel comfortable asking questions and so we can hunt down the answers and make sure you have the best treatment experience from day one.”

DAYBUE will be also come from an “exclusive pharmacy partner” that include clinical pharmacists on call 24/7 to answer questions, Teehan added.

Promising Pipeline

DAYBUE is the result of Acadia’s 2018 exclusive license agreement with Australia-based Neuren Pharmaceuticals Limited (ASX: NEU) for the development and commercialization of trofinetide for the treatment of Rett syndrome and other indications in North America.

Acadia is currently collaborating with Massachusetts-based Stoke Therapeutics in the development of gene therapy platforms that could address the underlying genetic issues that affect Rhett patients and similar rare diseases, Teehan said.

DAYBUE is the second drug that Acadia has seen through FDA approval. In 2016, Acadia launched NUPLAZID – the first commercial therapy for hallucinations and delusions associated with Parkinson’s disease. Acadia’s current pipeline includes a similar drug candidate that is in the same family as NUPLAZID, but for the treatment of psychosis symptoms in Alzheimer’s patients. That program is in Phase 1 development, but Acadia hopes “to move to broader clinical development before the end of this year,” Teehan said, adding that market for Alzheimer’s treatments “is five to ten the size as Parkinson’s.”

“We’ve learned a lot from our work in that therapeutic category and we look forward advancing that program as quickly as we can,” he said.

Acadia Pharmaceuticals Inc.

Founded: 1993
CEO: Steve Davis
Headquarters: San Diego Business: Drug development company focused on treating the negative symptoms of schizophrenia, Alzheimer’s disease psychosis and neuropsychiatric symptoms in central nervous system disorders.
Stock: ACAD (NADAQ)
Revenue: $517.2 million (2022)
Employees: 550+
Website: www.acadia.com
Notable: Acadia has two FDA-approved drugs that are first-ever treatments – DAYBUE for Rett and NUPLAZID for Parkinson’s.